Access for One, Access for All
April 22, 2024
After winning her own access battles, Madi Vanstone continues to fight for other Canadians with rare diseases
For more than half of her young life, Madi Vanstone, 22, has been fighting for access to life-changing treatments for cystic fibrosis (CF). Diagnosed with the condition at 8 months old, Madi had the opportunity to enrol in a clinical trial for a novel CF treatment called Kalydeco a decade later. Just one problem: after the trial ended, Madi could no longer receive the costly drug without paying for it. She and her mother spent two years lobbying the Ontario government before they agreed to cover it. In the meantime, Madi’s family, supported by fundraising efforts in her community, had to cover a good portion of the drug’s $300,000 annual cost.
Then along came Trikafta, a life-changing treatment that helps about 90 percent of people with cystic fibrosis. “My mom and I immediately hopped on that battle,” Madi recalls. Once again, it took years before the government agreed to reimburse the medication for most people with CF.
Having devoted many hours to raising awareness of rare diseases in Canada, Madi cheered when the federal government announced a national rare disease strategy in 2023. What’s still missing, she says, is a plan for using the funds. As she sees it, “people in government should be creating roadmaps to get the funds to patients and designing coverage plans for patients.”
“I’m happy to take up the fight for access so people with rare disease can all live our lives.”
Throughout her years of advocacy, Madi has forged strong ties with the Canadian Organization for Rare Diseases (CORD), so it came as no surprise when the group invited her to present the awards on 2024 Rare Disease Summit Day. “I had planned to stay home that evening and dote on my new puppy, but I would do anything for CORD so I was happy to step in,” she says. “It was great to see people getting recognized.” Madi herself appeared on a list of a dozen “changemakers” in a 2023 campaign called I Am Number 12, launched by a group of rare disease organizations and Takeda Canada.
Madi takes every opportunity to thank pharmaceutical manufacturers who develop products for people like herself, and encourages them to “keep thinking of patients as part of your team.” Recalling how cystic fibrosis affected “my school, my social life, everything,” she aspires to a career in patient advocacy. “While we shouldn’t have to fight for access, I’m happy to take up the fight so people with rare diseases can all live their lives.”